Boehringer and partners set sights on cystic fibrosis gene therapy

Deal builds on 17 years work in UK academic consortium

Potentially curing people with cystic fibrosis by inserting a healthy gene directly into their lungs is the goal of a new research alliance.

Boehringer Ingelheim has today unveiled its partnership with the UK Cystic Fibrosis Gene Therapy Consortium (GTC)—consisting of three top universities - Imperial College London and the Universities of Oxford and Edinburgh— plus technology transfer company Imperial Innovations, and cell and gene therapy drug delivery specialists Oxford BioMedica (OXB).

The UK-based GTC linking up with the German pharma company is an eye-catching development in cystic fibrosis, an area the consortium has been working on for no less than 17 years.

The field has been opened up by Vertex and its small molecule (pill-based) therapies Kalydeco (launched in 2012) and Orkambi (2015), as these are first treatments to tackle the underlying cause of the disease, a faulty CFTR protein.

The consortium aims to go one better than treating the protein, and replace the faulty mutated gene with a healthy copy, delivered straight to the lungs in a lentiviral vector.

While Vertex’s treatments can only slow the progress of the disease, a CF gene therapy could potentially provide a cure for the disease – if a number of technical obstacles can be overcome, most notably the huge drug delivery challenge of making an inhalable gene therapy.

"The GTC believes that this partnership provides CF patients with the optimal chance to establish gene therapy as routine clinical practice, relevant to all patients, irrespective of their mutation status, and in due course to both prevent lung disease as well as treat established problems,” said Imperial College's Professor Eric Alton, co-ordinator of the UK Cystic Fibrosis Gene Therapy Consortium. “We would like to take this opportunity to warmly thank all of our fundraisers who have supported us over many years,” he added.

For Boehringer Ingelheim, the alliance gives it entry into the gene therapy field, in which many pharma companies are now rushing to invest. Its history in respiratory medicine, including COPD drug Spiriva will help to tackle the drug development challenges.

“Through this collaboration, we are joining forces with some of the top talents in this disease space to propel treatment advances forward,” said Clive R. Wood, Ph.D., senior corporate vice president, Discovery Research at Boehringer Ingelheim.

Meanwhile Oxford BioMedica is a leader in the field of lentiviral vector manufacturing, techniques it has pioneered in producing two groundbreaking products, Novartis’ CAR-T therapy Kymriah and GSK/Orchard Therapeutic’s gene therapy Strimvelis.

Another advantage over Vertex’s products would be that an effective gene therapy could work for all patients – Orkambi is currently only licensed for patients with two copies of the F508del mutation, which accounts for around 45% of the total population.

The deal gives Boehringer Ingelheim exclusive global rights to develop and commercialise this lentiviral vector-based gene therapy for the treatment of cystic fibrosis, but its financial terms haven’t been disclosed.

Vertex is investing in its own ‘next generation’ treatments, and this includes a collaboration in the gene-editing field with CRISPR first launched in 2015. However this approach recently hit a setback when the FDA put a planned clinical trial of sickle cell candidate CTX001 on hold.